RNAi COMPOSITIONS AND METHODS FOR SILENCED GROWTH FACTOR RECEPTOR-BOUND PROTEIN 14 (GRB14) IN THE LIVER

AR135530A1Pending Publication Date: 2026-09-30ALNYLAM PHARMACEUTICALS INC
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Patent Information

Application Number
ARP20250100750
Authority / Receiving Office
AR · AR
Patent Type
Applications
Current Assignee / Owner
Priority Date
2025-02-24
Filing Date
2025-03-21
Publication Date
2026-09-30
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Claims

The disclosure relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the GRB14 gene, as well as methods for inhibiting GRB14 expression and methods for treating subjects who would benefit from reduced GRB14 expression, such as subjects suffering from a GRB14-associated disease, disorder, or condition, such as diabetes, using such dsRNA compositions.

1. A double-stranded ribonucleic acid (dsRNA) agent for inhibiting the expression of growth factor receptor-binding protein 14 (GRB14) in a cell, wherein said double-stranded RNA agent comprises a sense strand and an antisense strand forming a double-stranded region, wherein said antisense strand comprises a region of complementarity with an mRNA encoding GRB14 comprising at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any of the antisense sequences listed in either Tables 2-7 or 15-16.

51. A cell containing the dsRNA agent according to any of claims 1-50.

52. A vector encoding at least one strand of the dsRNA agent according to any of claims 1-50.

53. A pharmaceutical composition for inhibiting the expression of the growth factor receptor protein 14 (GRB14) gene comprising the dsRNA agent according to any of claims 1-50.